AAV.GTX-GLUT1

AAV.GTX-GLUT1 is an adeno-associated virus (AAV) vector-based gene therapy designed for the treatment of glucose transporter type 1 (GLUT1) deficiency syndrome, also known as De Vivo disease. The therapy utilizes an AAV vector to deliver a functional copy of the SLC2A1 gene, which encodes the GLUT1 protein, directly to the central nervous system via intrathecal administration. GLUT1 is the primary transporter responsible for moving glucose across the blood-brain barrier; its deficiency leads to low glucose levels in the cerebrospinal fluid (hypoglycorrhachia), resulting in refractory seizures, developmental delays, and movement disorders. By restoring GLUT1 expression, AAV.GTX-GLUT1 aims to normalize brain glucose metabolism and improve neurological and functional outcomes. The program is being evaluated in clinical trials supported by the Japan Agency for Medical Research and Development (AMED).